Clinical trial: New drug combination reduces anemia in rare blood disorder

Medical Xpress February 4, 2025
Olivia Dimmer, Northwestern University

A combination treatment has shown promise for treating a rare blood disease, according to a clinical trial published in the journal Blood.

Paroxysmal nocturnal hemoglobinuria, or PNH, is a life-threatening blood disorder that causes the body to destroy red blood cells within blood vessels, also called intravascular hemolysis (IVH). This often leads to severe anemia and other complications that can impact quality of life and survival of affected patients.

Previous studies have demonstrated that the drugs ravulizumab and eculizumab—which work by blocking complement component 5 (C5), a protein that plays a key role in cell-killing processes—were effective in controlling PNH by controlling IVH. However, a subset of patients receiving those drugs remained anemic because of a different process related to...